JP2021520231A - シュタルガルト病の処置のための組成物及び方法 - Google Patents

シュタルガルト病の処置のための組成物及び方法 Download PDF

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JP2021520231A
JP2021520231A JP2021504130A JP2021504130A JP2021520231A JP 2021520231 A JP2021520231 A JP 2021520231A JP 2021504130 A JP2021504130 A JP 2021504130A JP 2021504130 A JP2021504130 A JP 2021504130A JP 2021520231 A JP2021520231 A JP 2021520231A
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sequence
abca4
seq
nucleic acid
aav vector
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Inventor
グレゴリー エス． ロビンソン，
グレゴリー エス． ロビンソン，
ミシェル イー． マックレメンツ，
ミシェル イー． マックレメンツ，
ロバート マクラーレン，
ロバート マクラーレン，
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Oxford University Innovation Ltd
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Oxford University Innovation Ltd
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    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/44—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor
    • C12N2840/445—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor for trans-splicing, e.g. polypyrimidine tract, branch point splicing

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JP2021504130A 2018-04-05 2019-04-05 シュタルガルト病の処置のための組成物及び方法 Pending JP2021520231A (ja)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201862653085P 2018-04-05 2018-04-05
US62/653,085 2018-04-05
US201862765181P 2018-08-16 2018-08-16
US62/765,181 2018-08-16
US201862734479P 2018-09-21 2018-09-21
US62/734,479 2018-09-21
US201862774004P 2018-11-30 2018-11-30
US62/774,004 2018-11-30
PCT/US2019/026061 WO2019195726A1 (en) 2018-04-05 2019-04-05 Compositions and methods for the treatment of stargardt disease

Publications (1)

Publication Number Publication Date
JP2021520231A true JP2021520231A (ja) 2021-08-19

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JP2021504130A Pending JP2021520231A (ja) 2018-04-05 2019-04-05 シュタルガルト病の処置のための組成物及び方法

Country Status (5)

Country Link
US (1) US20210147870A1 (zh)
EP (1) EP3775230A1 (zh)
JP (1) JP2021520231A (zh)
TW (1) TW202006140A (zh)
WO (1) WO2019195726A1 (zh)

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2986635B1 (en) 2013-04-18 2018-10-03 Fondazione Telethon Effective delivery of large genes by dual aav vectors
EP3472328A1 (en) * 2016-06-15 2019-04-24 Oxford University Innovation Limited Dual overlapping adeno-associated viral vector system for expressing abc4a

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Publication number Publication date
EP3775230A1 (en) 2021-02-17
TW202006140A (zh) 2020-02-01
US20210147870A1 (en) 2021-05-20
WO2019195726A1 (en) 2019-10-10
WO2019195726A9 (en) 2020-11-05

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